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US 8,785,168 B2Granted
Formulations for lysosomal enzymes
AssigneeBiomarin Pharmaceutical Inc.
InventorsJonathan LeBowitz, Byeong Chang
Life of this patent
Estimating the term…
Abstract
The present invention provides improved formulations for lysosomal enzymes useful for enzyme replacement therapy. Among other things, the present invention provides formulations that preserve or enhance the stability and/or efficacy of a lysosomal enzyme such as acid alpha-glucosidase.
Claims21
A pharmaceutical composition for treating Pompe disease comprising a recombinant human acid alpha-glucosidase (GAA) in a formulation comprising:
- a) a buffering agent selected from the group consisting of histidine, sodium acetate, citrate, phosphate, succinate, Tris and combinations thereof;
- b) a stabilizing agent selected from the group consisting of sucrose, arginine, sorbitol, mannitol, glycine, trehalose and combinations thereof;
- c) a tonicity modifier selected from the group consisting of glycine, sorbitol, sucrose, mannitol, sodium chloride, dextrose, arginine and combinations thereof;
- d) a bulking agent selected from the group consisting of sucrose, mannitol, glycine, sodium chloride, dextran, trehalose and combinations thereof;
- and
- e) a poloxamer, wherein the poloxamer is Pluronic® F-68 at a concentration ranging between 0.001% and 0.2%,
- wherein the pH ranges from approximately 4.0 to 7.0.
4 more elements
A pharmaceutical composition comprising a lyophilized mixture of a recombinant human acid alpha-glucosidase (GAA), sodium citrate, mannitol, trehalose, and Pluronic® F-68.
Dependencies are inferred from the claim text.